Australia Pompe Disease Drugs Market Analysis

Australia Pompe Disease Drugs Market Analysis


$ 3999

Australia Pompe Disease Drugs market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 - 2030. The market for Pompe disease Drugs is growing due to increase in awareness about the disease , better diagnosis, advancements in treatment options and increase in research and development activities by different pharmaceutical companies and research organizations. Sanofi Genzyme ,BioMarin Pharmaceutical Inc., Avrobio Inc., Amicus Therapeutics Inc. are the key market players operating in global pompe disease market.

ID: IN10AURD106 CATEGORY: Rare Diseases GEOGRAPHY: Australia AUTHOR: Gaurav Bhongale

Buy Now

Australia Pompe Disease Drugs Market Analysis Summary

Australia Pompe Disease Drugs Market is valued at around $17.6 Mn in 2022 and is projected to reach $29.13 Mn by 2030, exhibiting a CAGR of 6.5% during the forecast period 2023-2030.

A genetic mutation that alters the function of the enzyme known as acid alpha-glucosidase (GAA) is what causes Pompe's illness. This enzyme converts glycogen, a kind of sugar that is stored in the body, into glucose, which the body can utilise as fuel. GAA enzyme activity is low or nonexistent in Pompe illness, which causes the buildup of glycogen in many organs, especially muscles. The symptoms of this disease are Muscular weakness, hypotonia, respiratory problems, fatigue and an enlarged heart. Enzyme replacement therapy, which entails giving patients recombinant acid alpha-glucosidase (GAA), the missing enzyme, is the main treatment for Pompe disease. ERT tries to control the disease's symptoms and lessen glycogen buildup. Companies that have created and marketed enzyme replacement therapy for Pompe disease are the major players in the market for medications for the condition and are also projected to grow in future.

Market Dynamics

Market Drivers

Improve in diagnosis, advancements in treatment options, development of Enzyme replacement therapy drugs and increase in research and development activities by different pharmaceutical companies and research organizations are all market growth drivers.

Market Restraints

limited patient population, high treatment costs, and regulatory complexities associated with the approval of drugs all act as market growth restraints.

Key players

Sanofi Pfizer Shire plc BioMarin Pharmaceutical Inc. Catalyst Biosciences uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals Intercept Pharmaceuticals Novartis

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For Australia Pompe Disease drugs market

By Treatment type

  • Enzyme Replacement Therapy (ERT)

By Age of Onset

  • Infantile Pompe Disease
  • Late-Onset Pompe Disease

By End-Users

  • Hospitals and clinics
  • Speciality Centers
  • Homecare

By Distribution Channels:

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies
  • Others

Methodology for Database Creation

Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.​

Additionally, we provide a comprehensive list of Key Opinion Leaders (KOLs) based on your requirements. Our curated list captures various crucial aspects of the KOLs, offering more than just general information. Whether you're looking to boost brand awareness, drive engagement, or launch a new product, our extensive list of KOLs ensures you have the right experts by your side. Covering 30 countries and 36 specialties, our database guarantees access to the best KOLs in the healthcare industry, supporting strategic decisions and enhancing your initiatives.

How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:

  • Analyzing historical proprietary data collected from multiple projects.
  • Regularly updating our existing data sets with new findings and trends.
  • Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

  • Searching through academic conferences, published research, citations, and social media platforms
  • Collecting and compiling diverse data to build a comprehensive and detailed database
  • Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

  • Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
  • Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
  • Continuously refreshing our database to ensure that the information remains current and reliable.
  • Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

  • Merging historical data from secondary research with real-time data from primary research.
  • Conducting thorough data validation and cleansing to remove inconsistencies and errors.
  • Organizing data into a structured format that is easily accessible and usable for various applications.
  • Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.

To request a free sample copy of this report, please complete the form below.


We value your inquiry and offer free customization with every report to fulfil your exact research needs.


Last updated on: 04 July 2023
Updated by: Dhruv Joshi

Related reports (by category)


Related reports (by geography)


subscribe to our newsletter
up